More Than Just a Number: Assessing The Value of Innovative Treatments

Value assessment frameworks are gaining traction in the United States and increasingly being applied in real-world settings. This shift in how we determine value in health care has the potential for long-lasting consequences on reimbursement decisions and access.

What’s more, the issues raised by such frameworks are growing increasingly complex, particularly with the introduction of curative specialized treatments, such as gene therapies. Unfortunately, headlines have been focused more on the costs of these treatments than the actual value and cures that they represent for patients.

Assessing the value of a treatment isn’t a new concept, but with the advent of these innovative curative medicines, there is a growing interest in determining how much our health care system is willing to pay. Several value assessment frameworks have been developed to assist payers with these decisions. Of those, the Institute for Clinical and Economic Review (ICER) has been the most prominent.

ICER, thanks in part to a significant injection of funding from the Laura and John Arnold Foundation, conducted 14 value assessments last year and the organization is on pace to match that number in 2019. However, it doesn’t mean that ICER should be the definitive word on how we view the value of a treatment.

ICER’s value assessments have some serious flaws that we hope will be addressed as it incorporates input from the public and updates its framework for 2020. We recognize that value assessments can be useful tools for health care decision-making, which is why our approach is to offer constructive feedback to organizations such as ICER and others working on value assessment.

It is critical that value assessment be patient-centered, and the National Pharmaceutical Council (NPC) published our own Guiding Practices for Patient-Centered Value Assessment to ensure the patient voice is included in these assessments. These guiding practices serve as a guidepost in framing our feedback.

In comments submitted to ICER, NPC recommended changes to improve ICER’s framework across 3 broad areas: changes to the framework itself, improvements in the way that assessments are conducted, and application of the framework beyond medicines to examine value across the entire health care system. The framework changes we recommend include a suggestion to use a collaborative and transparent model development process, similar to the approach that the Advisory Committee on Immunization Practices (ACIP) uses, in which a clinical expert working group can review both manufacturer and ACIP models side-by-side in a public meeting.

We also encouraged ICER to quantitatively include patient and societal factors in its assessments and to include this societal perspective as a base case model. We also underscored the need for ICER to evolve its thinking and incorporate more real-world evidence in the assessment process.

Some areas of the assessment process itself, too, deserve reconsideration. ICER should embrace full transparency by making its model publicly available to all stakeholders.

The organization should also present much broader results and context in its press releases and report-at-a-glance documents. Doing so can provide a fuller picture of the range of pricing scenarios and societal value, rather than implying a false sense of precision.

Finally, in order to achieve its goal of a “more just, efficient and more effective health care system,” ICER assessments must expand in focus to assess health care spending across the entire system. A lot of the discussions around value—and most of ICER’s assessments—have focused almost entirely on medicines, although drugs comprise just 16% of health spending in the United States.

ICER’s assessments must scrutinize the other 84%, as well. Medicines are a small portion of the US health care dollar, so ICER’s overall impact would be greater if it focused proportionately on other interventions, such as devices, surgery, and diagnostics.

We aren’t alone in raising concerns. Numerous patient groups have spoken out about the need for a more inclusive process in which patient voices have more weight when groups are assessing value.

Ultimately, it’s the patients who are affected most by value frameworks. Done well, such assessments can help guide patient access to high-value treatments. But done poorly, without fully taking the needs of patients into account, value assessments could be used as a mechanism that makes getting needed treatments harder.

Value assessment frameworks are expected to play a more pivotal role in decisions that affect future patient choice and access. Our challenge now is to ensure that existing frameworks, such ICER’s, adhere to value assessment guiding practices.

Complex and often difficult decisions are necessary as we seek to measure value in health care; however, taking steps now to ensure that the process is collaborative and grounded in best practices moving forward is in the best interest of patients.